Zokinvy (lonafarnib) is a prescription medication used to treat Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies (PDPL) in children aged 12 months and older. HGPS and PDPL are rare genetic disorders that cause premature aging.
Zokinvy is a farnesyltransferase inhibitor (FTI), which is a type of drug that blocks the activity of an enzyme called farnesyltransferase. Farnesyltransferase is an enzyme that plays a role in the production of a protein called progerin. Progerin is a defective protein that accumulates in the cells of people with HGPS and PDPL, and it is thought to be responsible for the premature aging symptoms of these diseases.
Zokinvy is given as a capsule twice daily with food. The dose is based on the patient’s height and weight.
How effective is Zokinvy?
In clinical studies, Zokinvy was shown to improve the quality of life and prolong the lifespan of children with HGPS and PDPL. In one study, children treated with Zokinvy had a median survival of 14.5 years, compared to a median survival of 8.2 years in children who received a placebo.
What are the side effects of Zokinvy?
The most common side effects of Zokinvy are:
- Nausea
- Vomiting
- Diarrhea
- Fatigue
- Headache
- Abdominal pain
- Decreased appetite
- Constipation
- Weight loss
Zokinvy can also cause more serious side effects, such as:
- Liver problems
- Kidney problems
- Allergic reactions
- Bone marrow suppression
Who should not take Zokinvy?
Zokinvy should not be taken by people who are allergic to lonafarnib or any of the other ingredients in Zokinvy. It should also not be taken by people with:
- Severe liver disease
- Severe kidney disease
- Pregnancy
- Breastfeeding
Important safety information
It is important to tell your doctor about all of the medications you are taking before starting Zokinvy. Zokinvy can interact with other drugs, such as certain antibiotics and anticonvulsants.
Zokinvy should be used with caution in people with liver disease, kidney disease, and diabetes.
Zokinvy can cause serious side effects, such as liver problems and kidney problems. It is important to monitor your liver and kidney function regularly while taking Zokinvy.
If you are considering taking Zokinvy, talk to your doctor about the risks and benefits of the medication.
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